Pharming Group N.V. announced that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending marketing authorization for Joenja® (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS) in adult and pediatric patients 12 years and older. A final decision by the European Commission is expected within approximately two months, which would make Joenja the first approved treatment for APDS in the European Union.
Key takeaways
The CHMP adopted a positive opinion recommending marketing authorization for Joenja® (leniolisib) for APDS treatment in patients aged 12 years and older.
A final European Commission decision on marketing authorization under exceptional circumstances is expected within approximately two months (Q2 2026).
If approved, Joenja would become the first approved treatment for APDS in the European Union, with centralized marketing authorization valid in all 27 EU Member States plus Norway, Iceland and Liechtenstein.
The positive opinion is based on results from a multinational, triple-blind, placebo-controlled, randomized Phase II/III clinical trial involving 31 patients aged 12 years and older.
Joenja is already approved and marketed in the United States and the United Kingdom for patients aged 12 years and older with APDS.
Leniolisib is currently under regulatory review for APDS treatment in Canada and several other countries, and is being evaluated in two Phase II clinical trials in primary immunodeficiencies with immune dysregulation.